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RECENSUS / INCEPTUS / ASPIRO Studies

Audentes Therapeutics is a biotechnology company committed to the development and commercialization of innovative new gene therapy treatments for people with serious rare diseases. They are a focused, experienced and passionate team driven by the goal of improving the lives of patients. Audentes Therapeutics is developing AT132 for the treament of X-Linked Myotubular Myopathy.

There have been several significant press releases on the progress being made by Audentes Therapeutics in moving the AT132 program forward and we are excited to share the following:

“It is important to note we cannot make any conclusions on the interim findings of the clinical trial until after all enrolled subjects are dosed and evaluated for the duration of the study, and the full scope of data is collected and analysed making conclusions about interim data may not accurately predict the full risk/benefit profile of an investigational product after all enrolled subjects are dosed and evaluated for the duration of the study, and the full scope of data is collected and analyzed, more complete information about safety and efficacy of this investigational gene therapy product will become available to the community.” released Audentes

AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), an ultra-rare, severe, debilitating disease that affects skeletal muscles in an estimated one in 50,000 newborn males worldwide, and is caused by mutations in the MTM1 gene

What is INCEPTUS? What is ASPIRO?

Both INCEPTUS and ASPIRO are clinical studies that are being or will be conducted by Audentes Therapeutics. The Audentes gene therapy product for XLMTM is called AT132. INCEPTUS and ASPIRO form part of the clinical development program for AT132. The overall aim of the clinical development program is to evaluate if AT132 is safe and effective in XLMTM, and to get the drug approved quickly in order to benefit patients with XLMTM as rapidly as possible. Audentes will collect the necessary data in the clinical studies and then, if the outcomes from the clinical studies are positive, file an application for potential approval to sell AT132 commercially with regulatory agencies around the world (e.g. FDA in the United States, and EMA in Europe).

What is RECENSUS?

Audentes Therapeutics has initiated its first study of X-Linked Myotubular Myopathy (XLMTM), entitled RECENSUS: A Medical Chart Review of Patients with XLMTM. RECENSUS is a Latin word which means to review and investigate. The goal of this study is to collect information from medical charts in order to characterize aspects of the condition and associated medical care for males with XLMTM.

This is a retrospective, non-interventional study, meaning there is a review of past care. It does not require visits to a study site or any medical procedures.

We hope that this study will contribute considerably to the understanding of XLMTM. If you are interested in having your or your child’s medical chart included in the study, please follow the link below for more information about the study and participating sites:

Those patients enrolled in the Begg’s registry do NOT need to do anything else. Their data is already being included in the study. Participation in this study does not influence the selection of patients for the clinical trial.